Research Horizons


Statins Effective in Treating Pulmonary Alveolar Proteinosis

Cholesterol-busting statins can effectively treat pulmonary alveolar proteinosis (PAP), a disease that causes air sacs in the lungs to clog with surfactant, according to new research led by Bruce Trapnell, MD, Director of our Translational Pulmonary Science Center.

Years ago, Trapnell and colleagues showed that PAP is linked to disrupted cell regulation by the molecule granulocyte-macrophage colony stimulating factor (GM-CSF). On Aug. 7, 2018, Trapnell and first author Cormac McCarthy, MD, PhD, reported in Nature Communications that the disruptions caused by GM-CSF reduce the ability of macrophages to process and clear out cholesterol. This contributes to the accumulation of surfactant that causes PAP and hinders breathing.

The discovery “will change thinking in the PAP field,” Trapnell says. “Now that we know cholesterol in macrophages is a target for therapeutic development, repurposing statins is a straightforward pharmacological approach for treating people with PAP.”

The findings are expected to lead to a larger clinical trial to test statin therapy.

Publication Information
Original title: Statin as a novel pharmacotherapy of pulmonary alveolar proteinosis
Published in: Nature Communications
Publish date: Aug. 7, 2018
Read the study

Research By

Bruce Trapnell, MD
Director, Transitional Pulmonary Science Center
Trapnell studies alveolar macrophage function; molecular techniques using gene knockout, transgenic and conditional gene expression mouse models and non-human primates, in vitro and in vivo viral gene transfer, and bone marrow transplantation.